PEARL (CML1624) = Asciminib in Early Treatment of CML [Spain]

What is the purpose of the study

This study will investigate the efficacy and the safety of asciminib at a dose of 80 mg once daily as single agent or 40 mg twice daily in combination with nilotinib 300 mg twice daily as early treatment for adult patients with newly diagnosed chronic-phase chronic myeloid leukemia (CML-CP).

What will happen during the study

Patients will receive either asciminib at a dose of 80 mg by mouth once daily as single agent (treatment arm A) or asciminib 40 mg by mouth twice daily plus nilotinib 300 mg by mouth twice (treatment arm B) for a minimum of 2 years. During the following 2 years, asciminib will be continued at the same dose in both arms; in the combination arm (arm B) the nilotinib dose will be reduced to 300 mg daily.

Patients maintaining a stable MR4 up to the end of the fourth year will discontinue the treatment.

Key inclusion criteria

This study includes patients of all sexes who:

• are aged 18 years or older.
• have a cytogenetic and molecular confirmed diagnosis of Philadelphia chromosome (Ph+) and BCR::ABL1 positive CML.
• are in early chronic phase, less than 3 months from diagnosis.
• have evidence of typical BCR::ABL1 RNA transcripts e13a2 or e14a2 (b2a2 or b3a2) at the time of study entry .
• have been treated with any tyrosine kinase inhibitor (TKI) for 30 days or less. Prior treatment with hydroxyurea or anagrelide is allowed.
• have an Eastern Co-Operative Group (ECOG) status of 0, 1 or 2.
• have adequate end organ function as defined in the study protocol.

Further criteria may apply. Please discuss these with your doctor or study staff.

Key exclusion criteria

This study does not include patients who:

• have CML in blast phase (BP) or in second chronic phase after previous BP, according to WHO criteria.
• have been treated with TKIs for more than 30 days.
• have a history or current diagnosis of heart disease indicating significant risk of safety for patients participating in the study.
• have a severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection).
• have had acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis.
• have a history of acute or chronic liver disease.
• have a history of other active malignancy within 2 years prior to study entry except for previous or concomitant basal cell skin cancer and previous carcinoma in situ treated curatively.

Further criteria may apply. Please discuss these with your doctor or study staff.

Estimated primary completion date

Where can I find additional information

You can find a study description in the US register ClinicalTrials.gov. This is a database provided by the U. S. National Institutes of Health.

Study centers / principal investigators

Spain

Valencia, Spain
Hospital Universitario La Fe Valencia
Contact : Elvira Mora

Barcelona
Hospital del Mar (Barcelona)
Contact: Patricia Vélez Tenza

Bilbao
Hospital Universitario Basurto
Contact: Fernando Marco de Lucas

Girona
Institut Català d’Oncologia Girona
Contact: Anna Angona Figueras

Granada
Hospital Virgen de las Nieves
Contact: José Manuel Puerta

Las Palmas De Gran Canaria
Hospital Universitario de Gran Canaria Dr. Negrín
Contact: Maria Teresa Gómez Casares

Madrid
Hospital Gral U. Gregorio Marañón
Contact: Santiago Osorio

Madrid
Hospital Universitario 12 de Octubre
Contact: Gonzalo Carreño Gómez-Tarragona

Madrid
Hospital Universitario La Paz
Contact: María Raquel de Paz Arias

Murcia
Hospital Clínico Universitario Virgen de la Arrixaca
Contact: Raul Perez Lopez

Salamanca
Complejo Asistencial Universitario de Salamanca
Contact: Magdalena Sierra Pacho

Valencia
Hospital Universitario La Fe Valencia
Contact: Elvira Mora